https://mirror.vjregenmed.com The Video Journal of Regenerative Medicine Tue, 04 Jan 2022 17:55:01 +0000 en-US hourly 1 https://wordpress.org/?v=6.5.2 https://d2xz56kaqxj8if.cloudfront.net/wp-content/uploads/2023/09/12102509/VJR-Favicon.png https://mirror.vjregenmed.com 32 32 https://mirror.vjregenmed.com/video/f-brn1smkkk-gnt0003-aav-gene-therapy-for-crigler-najjar-syndrome/ Thu, 23 Dec 2021 16:24:51 +0000 http://13.40.107.223/video/f-brn1smkkk-gnt0003-aav-gene-therapy-for-crigler-najjar-syndrome/ Lorenzo D’Antiga, MD, Hospital Papa Giovanni XXIII, Bergamo, Italy, describes the Phase I/II CareCN (NCT03466463) clinical trial evaluating GNT0003 therapy, an adeno-associated virus vector serotype 8 (AAV8) encoding hepatic UGT1A1, in patients with Crigler-Najjar syndrome (CN). In this dose-escalation study, no GNT0003-related serious adverse events were reported in the five patients treated. In patients treated with a higher dose, GNT0003 was demonstrated to be safe and restored UGT1A1 expression to levels allowing safe phototherapy withdrawal. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

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